Accelerating The Development of a Stem Cell Therapy For Vision Loss

March 8th, 2016 by FFB Canada

The race to develop a sight-restorative stem cell therapy is speeding up in Canada, thanks to a team of scientists and clinicians led by Dr. Valerie Wallace at the University Health Network in Toronto.

Dr. Wallace was recently awarded a prestigious Accelerator Fund from the Ontario Institute of Regenerative Medicine (OIRM) for her project Overcoming Central Vision Loss with Stem Cell Therapy and Rehabilitation. To strengthen the proposal, Dr. Wallace and her team successfully gathered a number of committed partners, including the FFB. Together, the funding partners pledged a total of $500,000 to support the project over the next year. The FFB is thrilled to partner with the OIRM to help fund Dr. Wallace’s project because it is gathering evidence that will enable a clear and effective path to the clinic. We are also very proud that the project team includes many exceptional vision researchers who have received funding from the FFB, such as the retinal stem cell pioneer, Dr. Derek van der Kooy.

The team will focus on collecting the critical evidence that is needed to demonstrate that vision can be restored to people who are blind by transplanting photoreceptors that have been derived from stem cells.

Although a number of clinical trials are currently testing stem cell therapies as a potential treatment for vision loss, these trials are not primarily aimed at sight restoration. Instead, the main goal is to prevent further vision loss. This is because the existing trials are transplanting a type of cell called retinal pigmented epithelial (RPE) cells, which provide support to the eye’s photoreceptor cells. The hope is that these transplanted RPE cells will prevent further vision loss by protecting photoreceptors, which are the eye’s light-sensitive cells. There are two kinds of photoreceptors: cones and rods. Cone photoreceptors are needed for high acuity central vision and are necessary for reading. Rod photoreceptors are needed for peripheral vision and for seeing in low-light. In age-related macular degeneration (AMD), cones are the cells that are lost. In retinitis pigmentosa (RP), rods are the cells that are lost first. In late-stage RP, the cones begin to degenerate as well.

Dr. Wallace’s team is developing cone photoreceptor transplantation as a treatment for blinding eye diseases that involve cone degeneration, including AMD and RP. The team is working toward two key milestones. First, they are building on Dr. Gilbert Bernier’s technique to generate cone photoreceptors that are a high enough quality to transplant into humans. Second, they are developing a transplantation technique to ensure that the new cone photoreceptors are able to function and restore vision. To do this, the team is drawing on the expertise of Dr. Molly Shoichet, a leader in the field of bioengineering, who has developed innovative methods to promote the survival and integration of transplanted cells.

Last week, we were very impressed after we watched the team present their results at a talk organized by OIRM. There is no doubt that this team is doing everything they can to accelerate the development of a new stem cell therapy for vision loss. Thanks to the FFB’s donors for helping to speed up the pace of research.

BB-RP

A question of timing.

Should I get a generic form of stem cell treatment for RP now or should I wait perhaps up to two years for the treatment outcomes of these trials?

I have an opportunity to receive stem cell therapy this year. I have savings to cover the cost of the treatment in California. It is HUSC using my own cells extracted from fat cells near the navel, isolating the SC with flow cytometry and then reintroducing them by injection.

The results in other patients have been interesting if varied, including some sight restoration and some slowing down of deterioration. However, because the stem cells are released generally in the body and expected to work on the areas most in need of repair, the effects can be unpredictable. In addition, their benefit can be eroded with time and compromised by sickness.

But this treatment doesn’t involve photoreceptor transplants discussed above. Nor is it any more or any less scientifically proven.

I understand that timing really matters because RP is a degenerative disease – this means that there is urgency to do something before things get worse. But, if you still have vision, there is a risk that you could lose this remaining vision by undergoing an un-tested “therapy” – I hesitate to use the word “therapy” because these are really just experiments, not therapies.

Lots of scientists and clinicians are working around the world to develop something that works. We strongly recommend that you only participate in clinical trials that have undergone the proper approval process.